Haemophilia outcome
measures: Industry’s view
EHC Round Table of Stakeholders
‘Outcome measures in haemophilia’
Brussels, Belgium
November 28 2016
ABOUT THE SPEAKER
•
•
•
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Jason Booth, MPH
Associate Director, Global HEOR Haematology
Shire
US
DISCLOSURES
Conflict
Disclosure - if conflict of interest exists
Employee
Shire
We need to better understand and
use health outcome information in
haemophila
• Expectations of people with haemophilia are increasing all over the
world and participation in social, work and physical activities is
improving1
• Annualised Bleed Rate (ABR) is significantly reduced with
prophylaxis compared to on demand treatment2 however ABR
doesn’t reflect all disease impacts
• As a result ABR has limited utility in measuring further improved
outcomes and current Health-Related Quality of Life measures are
of limited use outside a research setting1
• With new therapies emerging it is important to develop an
individualized view of the impact of therapy and, with support from
payers, to use this information to improve standards of care
1.Recht, et al. Recognizing the need for personalization of haemophilia patient-reported outcomes in the prophylaxis era. Haemophilia. (2016), 1–8
2. Ljung, et al. The current status of prophylactic replacement therapy in children and adults with haemophilia. British Journal of Haematology (2015) 169, 777–786
New measures should be practical,
sensitive and complement existing
measures
Complementary to
standard outcomes,
such as ABR1
Aligned with
measures
meaningful to
payers3
Encourage and
support best
practice in patientcentric care1
Sensitive to small
changes that are
meaningful to
patients1
Practically useful in
clinical care AND as
a research tool1
1. Recht, et al. Recognizing the need for personalization of haemophilia patient-reported outcomes in the prophylaxis era. Haemophilia. (2016), 1–8
3. Berger et al. Securing reimbursement for patient centered haemophilia care: major collaborative efforts are needed. Haematologica (2016); 101 (3)
Goal Attainment Scaling (GAS) is an
established approach to quantifying individual
treatment goals1
• Shared decision making in the process of setting
goals and appropriate therapy is essential for
effective patient-centric care5
• GAS - A standardized approach to individualized
(patient-centered) goal monitoring, that is often
done informally in clinical care1
• Engages the patient/caregiver in a dialogue about
what is important to him/her
• Assesses extent to which a patient is able to
meet/exceed their individual goals and also assess
this consistently across a population4
1. Recht, et al. Recognizing the need for personalization of haemophilia patient-reported outcomes in the prophylaxis era. Haemophilia. (2016), 1–8
4. Turner-Stokes, et al. Goal attainment scaling (GAS) in rehabilitation: a practical guide. Clinical Rehabilitation 2009; 23: 362–370
5. Barry et al. Shared Decision Making — The Pinnacle of Patient-Centered Care. N Engl J Med (2012) 366;9: 780-781
Standard Steps for GAS4
Define and select a patient identified goal
Define goal achievement (targeted / expected outcome)
Describe the baseline status
Define the better and worse outcomes to complete the scale
Measure goal attainment at designated follow-up interval(s) using
the scale developed
4. Turner-Stokes, et al. Goal attainment scaling (GAS) in rehabilitation: a practical guide. Clinical Rehabilitation 2009; 23: 362–370
Standard Approach: 5-Point
Scale6
Goal
Baseline
6. Figure from http://clik.dva.gov.au/rehabilitation-policy-library/15-goal-attainment-scaling/151-introduction-goal-attainment-scaling Accessed Nov 2016
A GAS for haemophilia is
currently in development7
Goal Attainment
Scaling for Life –
Hemophilia
(GOAL-Hem) will
be a hemophiliaspecific menu of
patient-centered
goal areas for use
in clinical practice
and research8
7. https://clinicaltrials.gov/ct2/show/NCT02674997 Accessed November 2016
8. Recht, et al. Goal Attainment Scaling for Life – Hemophilia (GOAL-Hēm): An Innovative Patient-Reported Outcome Measure. Poster presented at WFH
World Congress, Orlando, USA (2016)
Constructing goals with the
GOAL-Hem – Example
Goal: Start exercise program to improve joint health
• (+2) I have joint problems that bother me most days: however, I
am able to manage physical/emotional toll with exercise 5x week
and able to stand on my feet >30 min without severe pain
• (+1) I have joint problems that bother me most days: however, I
am able to manage physical/emotional toll with exercise 3x week
and able to stand on my feet >20 min without severe pain
• GOAL (0): I have joint problems that bother me most days:
however, I am able to manage physical/emotional toll with
exercise 2x week and able to stand on my feet >15 min without
severe pain
• BASELINE (–1): I have joint problems that bother me constantly:
however, it is difficult to manage physical/emotional toll with
physiotherapy and/or pain management
• (–2) I have joint problems that bother me constantly: however, I
am unable to manage physical/emotional toll with physiotherapy
and/or pain management and have no desire to do so
GAS scoring standardizes individual
goals across different patients
• Scores are effectively the sum of goal
attainment X the relative goal weights
transformed into a standardized (normally
distributed) measure (T-score)4
• Overall scoring returns summary score of 50
when all goals are attained (individual goal
attainment = 0)7
4
4. Turner-Stokes, et al. Goal attainment scaling (GAS) in rehabilitation: a practical guide. Clinical Rehabilitation 2009; 23: 362–370
7. https://clinicaltrials.gov/ct2/show/NCT02674997 Accessed November 2016
We need to better utilise existing and new
outcome measures to drive improved care
“Cigna stressed that a key factor in this
• Health systems should provide the
best possible health outcomes
success was the alignment of payer and
while utilising available resourcesmanufacturer
in the most incentives
efficient way
to support better
outcomes.” Neumann et al (2011)10
• If the right outcomes are measured and a procurement system is
centred around these, improved efficiency (i.e. outcomes for
resource used) could be achieved through personalized care9
Example of procurement on adherence and outcomes performance in diabetes10
Outcome measure
Glucose level reduction &
adherence
Discount if patients reduce glucose levels
Additional discount if patients are adherent

If outcomes are improved, the manufacturer (Merck) provides a discount to the payor (Cigna) at the end
of the year on their oral diabetes drug (Januvia and Janumet)

Outcomes measured are blood sugar levels (through A1C lab tests) and adherence (through claims data)
Results after the first year

Blood sugar levels dropped by 5%

Adherence increased by 87%
9. Gringeri, et al. An innovative outcome-based care and procurement model of hemophilia management. Expert Review Pharmacoeconomics & Outcomes Research,
(2016) 16(3), 337–345
12
10. Neumann et al. Risk-Sharing Arrangements That Link Payment For Drugs To Health Outcomes Are Proving Hard To Implement. Health Affairs 30, no. 12 (2011):
2329–2337
Procurement on price and volume does not
create right incentives to improve care and
value for money
In the current procurement paradigm two levers exist to address
efficiency and sustainability:
Current paradigm:
Price per IU
Reduce utilization
Reduce price

May only be possible if patients are
not optimally treated

One-off mechanism not addressing
long term growth

Risks worsening patient outcomes

Potentially reduces access to
innovation for patients
Future efficiency and sustainability requires a new approach
to procurement of haemophilia care
Outcomes data collection is critical to
measuring and improving system
efficiency
9. Gringeri, et al. An innovative outcome-based care and procurement model of hemophilia management. Expert Review Pharmacoeconomics & Outcomes
Research, (2016) 16(3), 337–345
Outcomes based procurement could
better align incentives across all
stakeholders
9. Figure from Gringeri, et al. Expert Review Pharmacoeconomics & Outcomes Research, (2016) 16(3), 337–345
Summary and Conclusions
• There is a need for more individualized
outcome measures including patient
treatment goals
• These should be complementary to existing
clinical and patient reported outcomes, and
useful in clinical practice and research
• New and existing outcomes could be used as a
basis for procurement to incentivize optimal
care in a financially sustainable way
EUBS/MG1/16-0087 November 2016